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Sodium oxybate
go.drugbank.com/drugs/DB09072ApprovedInvestigational[A18723] For safety reasons, sodium oxybate is a controlled substance only available through a restricted program in approved countries. … in May 2023.
Abiraterone
go.drugbank.com/drugs/DB05812ApprovedInvestigationalAbiraterone is a potent, irreversible, and selective inhibitor of 17 αhydroxylase/C17,20-lyase (CYP17), an enzyme expressed in testicular, adrenal, and prostatic tumour tissues, to regulate androgen biosynthesis … [L40968, L40193, L47740, L47745] and when used in combination with niraparib, BRCA2-mutated metastatic castration-sensitive prostate cancer (mCSPC).
Products: Abirateron 1A Pharma 500 mg - Filmtabletten, Abirateron 1A Pharma 1000 mg – FilmtablettenCerliponase alfa
go.drugbank.com/drugs/DB13173ApprovedInvestigational[L51329] CLN2 is a predominantly pediatric-onset neurodegenerative disease caused by a deficiency in the lysosomal enzyme TPP1, leading to progressive impairment in motor and cognitive function. … [A264354, A264369] As a recombinant human TPP1, cerliponase alfa is used as an enzyme replacement therapy to restore the levels of TPP1 in patients with CLN2.[L51339]
Categories: Hydrolytic Lysosomal N-terminal Tripeptidyl PeptidaseSynonyms: Immature tripeptidyl-peptidase IMigalastat
go.drugbank.com/drugs/DB05018ApprovedInvestigationalFabry disease is a rare, progressive genetic disorder characterized by a defective GLA gene that causes a deficiency in the enzyme alpha-Galactosidase A (alpha-Gal A). … where the agency determines that the drug if approved, would provide a significant improvement in treating, diagnosing or preventing a serious condition over available therapies.
Categories: Alpha-Galactosidase A (alpha-Gal A) Pharmacological ChaperonesRiociguat
go.drugbank.com/drugs/DB08931ApprovedInvestigationalRiociguat is a soluble guanylate cyclase (sGC) agonist approved in the USA, Europe and several other regions for patients with group I PAH (pulmonary arterial hypertension) in WHO FC II or III; and for … the treatment of patients with inoperable CTEPH (chronic thromboembolic pulmonary hypertension), or persistent/recurrent PH (pulmonary hypertension) after pulmonary endarterectomy in WHO FC II or III.
Synonyms: Methyl N-[4,6-Diamino-2-[1-[(2-fluorophenyl)methyl]-1H-pyrazolo[3,4-b]pyridin-3-yl]-5-pyrimidinyl]-N-methyl-carbaminateNavepegritide
go.drugbank.com/drugs/DB17824ApprovedInvestigationalAchondroplasia, the most prevalent skeletal dysplasia, is caused by a gain-of-function variant in the fibroblast growth factor receptor 3 (FGFR3), which leads to overactive downstream signaling that inhibits … [L56058,A275435] Navepegritide is a prodrug of active C-type natriuretic peptide (CNP) consisting of a CNP moiety transiently conjugated to two methoxy polyethylene glycol (mPEG) moieties via a proprietary
Gadoxetic acid
go.drugbank.com/drugs/DB08884ApprovedInvestigationalGadoxetic acid (gadoxetate) is a paramagnetic gadolinium-containing ionic linear contrast agent in which its salt form, gadoxetate disodium, is used for intravenous injection. … Ethoxybenzyl diethylenetriaminepentaacetic acid is the moiety that chelates with a gadolinium ion and forms a stable complex with it to make up the drug.
Categories: Compounds used in a research, industrial, or household settingCipaglucosidase alfa
go.drugbank.com/drugs/DB16708ApprovedInvestigationalby a deficiency in GAA. … in Pompe disease.
Rice bran
go.drugbank.com/drugs/DB14361ApprovedRice bran is a plant/plant extract used in some OTC (over-the-counter) products. It is not an approved drug.
Mixtures: Ari S-LinerRelacorilant
go.drugbank.com/drugs/DB14976ApprovedInvestigationalRelacorilant is a first-in-class, selective glucocorticoid receptor antagonist. … [L56089] Relacorilant was approved by the US FDA on March 25, 2026, for use in combination with [paclitaxel] in patients with certain platinum-resistant cancers.[L56093,L56089]